Long-term effectiveness, safety, and liver stiffness dynamics of PBC treatment with obeticholic acid in real-world

作者：Francesca Terracciani, Antonio De Vincentis, Daphne D’Amato, Laura Cristoferi, Alessio Gerussi, Pietro Invernizzi, Miki Scaravaglio, Ester Vanni, Daniela Campion, Anna Morgando, Vincenzo Valiani, Vincenzo Boccaccio, Filomena Morisco, Lorenzo Surace, Ilaria Cavalli, Guido Delle Monache, Federico Salomone, Donatella Ieluzzi, Debora Angrisani, Raffaella Tortora, Biagio Cuffari, Alessandra Moretti, Gerardo Nardone, Stefano FAGIUOLI, Mauro Viganò, G Vettori, Federica Cerini, Giancarlo Gimignani, Alberto Mattalia, Fabrizio Pizzolante, Nicoletta De Matthaeis, Cristina Rigamonti, Giulia Francesca Manfredi, Valentina Boano, Paola Begini, Ana Lleò, Francesca Colapietro, Riccardo Plebani, Domenico Alvaro, Rosanna Venere, Elisabetta Degasperi, Marta Borghi, Pietro Lampertico, Edoardo Giovanni Giannini, Sara Labanca, Raffaella Viganò, Federico D’Amico, Antonino Castellaneta, Francesco Squeo, Luca Cadamuro, Luigi Capodicasa, Marco Marzioni, Valerio Buzzanca, Guido Poggi, Alessandro Mussetto, Raffaele Cozzolongo, Francesco Losito, Gaetano Bertino, Maurizio Russello, Mariarita Cannavò, Paolo Scivetti, Maurizio Pompili, Annalisa Tortora, Grazia Anna Niro, Rosa Cotugno, Pietro Pozzoni, Alessia Riva, Luchino Chessa, Michela Miglianti, Giuseppe Cuccorese, Valeria Pace Palitti, Ludovico Abenavoli, Natalia M. Terreni, Teresa Zolfino, Olivia Morelli, Carlo Saitta, Silvia Casella, Adriano Maria Pellicelli, Maurizia Rossana Brunetto, B. Coco, Andrea Galli, Fabio Marra, Armando Curto, Annarosa Floreani, Nora Cazzagon, Paolo Rollo, Emanuela Bonaiuto, Loredana Simone, Luigi Muratori, Floriano Rosina, Marco Distefano, Elisabetta Capello, Valentina Bellia, Rodolfo Sacco, Giuliano Alagna, Leonardo Baiocchi, Lory Saveria Crocè, Chiara Assunta Ricci, Paolo Poisa, Antonio Izzi, Sara Boninsegna, Vincenza Calvaruso, Marco Carbone, Umberto Vespasiani Gentilucci, Giulia Di Pasquale, Paolo Gallo, Cossiga Valentina, Falbo Elisabetta, Tatonetti Riccardo, Musumeci Martina, Christou Chrysanthi, Paolo Villa, Eugenia Pesatori, Alessandro Loglio, Alessandro Conforti, Valentina Feletti, Alessandra Garriba, Evelise Frazzetto, Sara Alecci, Annalisa Ardiri, Martina Costanzo, Francesca Romana Ponziani, Francesca Fianchi, Niccolò Bina, Francesca Zani, Barbara Omazzi, Olga Falco, Jessica Piroddu, Anna Rocca, Paola Carnì, Giuseppe Grassi, Francesco Pezzato, Erica D'Ovidio, Matteo Peviani, Alessandro Bellia, Maria Cristina Sapere, Massimiliano Loreno, Masutti Flora, Fusaro Lisa, Riccardo Perbellini, Roberta Soffredini, Concetta Pitrone, Roberto Filomia, Alba Rocco, Pasquale De Crescenzo, Pasquale Piscitelli, Francesca Toffolon, Fabiana Marchetti, Alessandro Inno, Marco Guarracino, Giovan Giuseppe Di Costanzo, Nicola Alessi, Maria Cristina Neglia, Scudu Chiara · 发表于：JHEP Reports · 年份：2025 · DOI：10.1016/j.jhepr.2025.101448 · 被引用次数：5 · 研究领域：Liver Disease Diagnosis and Treatment、Liver Diseases and Immunity、Drug-Induced Hepatotoxicity and Protection

Background and Aims A number of studies have assessed short-term effectiveness and safety of obeticholic acid (OCA) in the real-world setting. We aimed to extend the knowledge on real-world OCA treatment effectiveness and safety by expanding sample size and follow-up, and by exploring liver stiffness measurement (LSM) changes over time. Methods The RECAPITULATE project involves centres belonging to the "Italian-PBC-registry" and/or the "Club-Epatologi-Ospedalieri" (CLEO) PBC working group. Effectiveness was evaluated as biochemical response according to PBC-OCA-International-Study-of-Efficacy (POISE) and normal range (NR) criteria (normal ALP/ALT/bilirubin). Safety was assessed as incidence of de novo /worsening pruritus and discontinuation rate/causes. Available LSMs were also captured. Results We included 747 patients from 66 Italian centres [58 years; F/M 88%/14%, median follow-up 24 months (IQR 12-42), 28% with cirrhosis, and 14% with autoimmune hepatitis (AIH)/PBC overlap]. Probabilities of POISE and NR response increased from baseline up to 57% and 20% at the 42nd month, respectively. They were lower in cirrhosis ( p =0.02 and p =0.004 for POISE and NR), while not different between AIH/PBC overlap and PBC ( p =0.8). Overall, 130 patients (17%) discontinued treatment, mainly due to pruritus (36.9%), while 28.5% did so after developing hepatic events. Discontinuation rate was higher in cirrhosis ( p <0.001). LSM was available in 573 patients (∼77%), of whom 255 had multip...